USA – FDA Revises Draft Guidance on Rare Diseases

The US Food and Drug Administration (FDA) has updated a 2015 draft guidance on rare diseases, with new information gleaned since the original draft was released.

Among the revisions to the 24-page draft guidance, according to FDA, are updates to the natural history studies section, the inclusion of issues for evaluating and validating biomarkers as surrogate endpoints, a discussion of additional flexibility on the non-clinical aspects of the evaluation of novel drug compounds, the addition of a section describing the evaluation of safety questions and added information on changes to drug substance or drug product manufacturing process with clarification on areas of additional flexibility.

“The revised draft guidance issued today discusses select issues commonly encountered in rare disease drug development. While similar issues often also come up for common diseases, they’re frequently more difficult to address in the context of a rare disease for which there’s often limited medical and scientific knowledge,” FDA Commissioner Scott Gottlieb said.